Survey results: why are MSers such risk takers?

I had no idea that MSers were such risk takers. #MSBlog #MSResearch

"Live and learn something everyday. I am bowled over by the preliminary results of haemopoietic stem cell transplant (HSCT) survey. I had no idea how willing MSers are to accept unlicensed treatments, with evidence that is essentially multiple case series, rather than the gold-standard randomised controlled clinical trials. Not to mention the risks they are prepared to take. We will need to follow this up with some high quality qualitative research in the UK; but if the message holds we will need to explore how we can start to offer this treatment in the context of a clinical trial. We will need to show that HSCT is effective and more importantly a cost-effective alternative to licensed treatments."

"If you want to contribute to the survey it is still open."

Some of the comments from the survey so far
  1. Anecdotal reports suggest HSCT can halt progression in PPMS and a trial would help discover the truth.
  2. There's a difference in having a life to being alive. For that reason, I'd take a 1-in-20 risk of dying at the outset of MS if that's a 19-in-20 chance at a normal life.
  3. I was dx'd at 30 years old. All my grandparents lived into their late 80s/90s. The prospect of 60 years of terminal decline, unemployment & financial destitution, relationship/family issues, chronic pain, progressive disability, dementia, and general despair is not for me.
  4. If there was a treatment with 50/50 chance of cure or death, i'd still take it over the propsect of 60 years of drawn out misery.
  5. Stop selling us such high risk, please.
  6. I think I would opt for meyoblative HSCT if I had highly active disease since there is enough data in my mind that it is the most effective therapy out there.
  7. Please enable self-funded international patients to be treated off-trial.
  8. I don't think you are too hung over on aggressive treatments or a laggard on HSCT. I think you are very sensible in your approach. My neurologist also thinks like you. He knows we will have little to no options once my disease becomes progressive and wants to stop it on it's track right NOW!
  9. Having a wife who "HAD" CIDP and saw the amazing results of HSCT for 80% + of people getting HSCT for all the autoimmune conditions treated by Dr Burt Chicago I as a patient would 100% want HSCT.
  10. I have PPMS and had HSCT with great results in Moscow with Dr Fedorenko. So pleased and grateful that this option was available to me.
  11. A bit academic for me as unfortunately I have SPMS. 
  12. I did HSCT with Dr. Richard Burt one year ago at Chicago Northwestern. HSCT stopped my MS after Tysabri had failed. 
  13. I fly in exactly 4 weeks to Moscow to be treated for SPMS with HSCT. I tried in this country with both Dr Silber at Kings and Prof Sharrack at Sheffield Hallamshire. I think we can safely say the UK Neurologists are too conservative! I did not want miracles; just to have the disease stopped in it's tracks. I have contributed 12% of my salary to the NHS for over 30 years and now I will be spending my life savings ($40,000) on being treated in Vladimir Putin's hospitals!
  14. If my MS progressed to be more of a burden on my life I would likely be prepared to take a higher risk of dying from a treatment.
  15. The level of risk (ie the risk of dying) acceptance is not constant: the older my children are growing, the higher the level of acceptance will be.
  16. As protocols evolve and increase the safety of the procedure HSCTs could provide a more cost effective treatment in the long term.
  17. The severity and progression of my MS would definitely dictate the risk I would be willing to take. Sometimes the risk of dying would be worth it if it meant that ridding this terrible disease and pain might end with treatment. Pain, whether physical or mental is tough to live with and I think patients should be given the choice. Its their body and their suffering they have to live with, not the doctors or lawmakers. 
  18. As protocols evolve and increase the safety of the procedure HSCTs could provide a more cost effective treatment in the long term.
  19. Well HSCT is a better option than euthanasia. 
  20. Why am I only now being made aware of Ocrelizumab ? My walking over the past month has deteriorated from 500 metres to 100 metres before needing a break.
  21. From the perspective of having grumbling but progressive MS rather than highly active MS i am not sure my opinion is valid. I find my slowly diminishing function frustrating and upsetting but I can adapt. If I had highly active MS I am sure I would want aggressive treatment, though 1:200 mortality risk seems quite a big risk still. 
  22. No chemo no cure?
  23. HSCT would be my treatment of choice and I will try to fund it myself should my MS start to kick off. Reason being I am young and have a life ahead of me. I want to live life as much as possible. Long term stats for MS are grim. HSCT is in my opinion the best change of curbing MS and all the destruction and lost dreams it brings. The low risk of death does not worry me but a prolonged period of severe and end stage MS does. 
  24. I am confused at how HSCT is safer (has less autoimmune problems) if it uses chemotherpay (such as alemtuzumab) to destroy bone marrow. 
  25. What about the genetic component of MS that was discussed at A/ECTRIMS in Boston? If a person's own stem cells are used aren't they at the same risk for MS before and after the therapy?
  26. The changes that happen otherwise healthy young adults who are diagnosed with MS is to me, worse than death- it is death by 1000 little 'losses' overtime that you 'get' to observe happen to your body without your input or consent. You know you will eventually loose most function and die an early unhealthy death to this disease- taking a chance on the front end to maintain your 'selfhood' seems a small risk compared to loosing everything slowly.
  27. I think the more options available to people, the better. I don't see HSCT as being different to any other type of treatment where the MSer and treating neurologist have to weigh up the risks and benefits. The trial results quoted on this blog do look very impressive. 
  28. The benefits far outweigh the risks! 
  29. MS is a terrible, debilitating, life-sentence of a disease. If HSCT slows it down or stops it, it should be offered. HSCT is a safe cancer treatment that has been around for many years.
  30. MS is known to be auto-immune. Therefore, HSCT, by ablating some or all of the immune system, will provide benefit for all types of MS. Even progressive.
  31. I think all effective therapies should be approved and allow doctors and patients to make the call on the risk-benefit trade-off. While HSCT appears to be pretty effective, I don't feel like I've seen good science on it, and so it seems more similar to CCSVI than the pharmaceuticals. However, I would be willing to take on fairly high risk to cure MS.
  32. What risk am I taking by not pursuing HSCT now that I am 54, healthy and don't have any current medical issues. What issues do I face by using Gilenya for 4+ years.
  33. In my opinion NIHR should fund a trial comparing HSCT to alemtuzumab, but not a randomized one. There are enough-patients who themselves would choose alemtuzumab because of the lower short term risk, and there certainly are many enough who wants HSCT. Therefore the trial hasn't at all have to be randomized (make a protocol with suitable inclusion and exclusion criteria). It is mandatory that besides using NEDA (number of relapses after treatment, EDSS after treatment and plaques and contrast enhancements on MRI) you also have to use quality of life (QoL) as an endpoint, for instance SF-36. For all ms-patients quality of life is greatly decreased and each day because of fatigue, pain, cog fog, bad sleep etc., and it is incredible that studied on DMDs - practiacally all of them - haven't applied QoL as an endpoint (but then, practically all the DMDs reduces QoL even more in addition). Therefore in a non-inferiority trial when you define a noninferiority margin and include a noninferiority hypothesis, you have to also include QoL as an endpoint!!!
  34. Besides, the NIHR should fund the same type of study for patients who have not had ms for a long time, and without highly-active ms (frequent relapses). The absolute majority of ms-patients are in this category, and their quality of life is low (it is mandatory for you to apply quality of life in all studies on DMDs and HSCT, and as Burt points out you haven't because DMDs don't improve QoL), and most of them have a dismal prognosis ending up out of work and in the long term with a high EDSS.
  35. I think HSCT is not only the best avalaible option to MSers with highly-active MS but also for
  36. PPMSers . You said that Ocrelizumab could be effective for PPMSers so I guess a more radical approach will be more effective.
  37. Anyway as today PPMSers have no a single one aproved DMT ,HSCT remain the best and only option.
  38. Have had non myelo in Russia Feb 14. EDSS of 4 now EDSS of 2.
  39. Alemtuzumab is not as effective as HSCT. I dont want to go through a procedure that does that comes with secondary concern for thyroid disease. I'd rather go through a process that halts the progression of MS and get on with the rest of my life. As the HSCT process is continued and refined. I believe the mortality rate will continue to decline.
  40. For those of us with RRMS that is transitioning and for those with PPMS or SPMS who are deteriorating noticeably HSCT is an excellent option as it arrests the disease progression in approx 80% (perhaps more now) of patients. It seems the protocol used in Russia is particularly effective on the more progressive types of MS. Cyclophosphamide with Rituximab,I am actively looking to get BMT..or HSCT presently, before my disability prevents me from being able to work, where the govt will have to support me entirely. HSCT has got to be a better option than this as it works out MUCH cheaper if it stops the disease and prevents further drugs and allows me to support myself!! A clinical study such as the one that is already in place in Sheffield would take too long to implement. Equally it is outdated since Russia has proven that HSCT works for progressive patients in stopping the progression of the disease! I think that peer published papers and practices such as what is in place in Canada and other parts of the world should be reviewed and adopted. No time for more trials!
  41. There is not really anything to comment! Anybody really knows that the only reason you are not offering this treatment is the market system...
  42. Same story as with vitamin-D, no difference!
  43. HSCT is worth it at every step - from Quality of Life Improvements to Financial Cost to the State to undergoing a Short Sharp Shock for a massive gain at the end of it.
  44. St Bart's should be performing HSCT!
  45. My son had highly aggressive ms had HSCT is a life saver. He is doing great no medication
  46. I am booked to have HSCT is Moscow in April 2015. It is ridiculous I have to fly half way round the world for treatment. HSCT HAS been done before in Glasgow,Scotland but it is not a routine treatment. It is also done in London and Sheffield UK buy with such narrow entrance criteria it is impossible for people to get on trial.
  47. Try living with MS? Then the medical profession would know about risk aversion for treatment compared to living with a degenerative condition. There is strong research and trial results supporting HSCT for MS. When will neurologists pay attention and look seriously at the evidence that is there? Oh, that's right. . .no ongoing revenue from people not taking drugs for the rest of their lives. Big pharma companies have way to much control of the medical profession. 
  48. I'd have this done asap if I could!! But I don't qualify for the trial & I don't want to leave the country!!! It's really very sad that this isn't available to everyone.
  49. I had HSCT for PPMS in June, 2014. My progression has stopped and I'm stronger, have better balance and I'm walking better then before the treatment. I have no regrets.
  50. I have been fighting for hsct for 2 yrs, I think a lot of the problem with approval is no nuro's will ok because it hasn't been okay ed by the FDA. It's been a treatment for 15 yrs in the United States and studied had by dr. Richard burt at northwestern university for autoimmune diseases and even longer over seas. And this same sort of procedure has been out for about 6 yrs in the US for cancer. Every body has there own way 2 heal and we finally found it! People who receive HSCT get there lives back and people with an autoimmune disease. Now have hope. But the chances for some people like myself of getting HSCT in time so it's more effective is so hard because you need to have your health insurance approve it. It's not like other trials where it free for patients to get the trial approved by the FDA. And a lot of us think it's because it's not a drug trial. Drug companies run the world it seems like. There is something out there that can really Help i have seen it first hand. why are we not even talking about this there are so many people I talk to at my MS infusions that have never heard of HSCT, but they have heard about all these new drugs that they will be taking for the rest of there lives. WHY IS THAT? HSCT is one treatment no more drugs. 
  51. HCST is the most current advanced procedure. The sheapest in long run and the safest in comparison to other treatments
  52. HCST is the most current advanced procedure. The sheapest in long run and the safest in comparison to other treatments
  53. Time is something few MSers who have had the illness for a long time can ill afford. HSCT is proven to work in sufficient numbers to war rent it being offered to those willing to take it.
  54. Am on waitlist for Russia- 2017-2018
  55. had hsct moscow 2013. 
  56. I received HSCT at Heildelber
  57. The 1-2% recent TRM is precisely what is seen in new HSCT protocols. I, for one, would advocate for the choice to treat very early on without the onerous requirements of DMD failures, none of which have shown any durability in preventing or halting progression. I had myeloablative HSCT just completed after 12 years of diagnosis and realize I still may never be able to walk right again. Not a single of my neurologists even mentioned HSCT. My quality of life is now destroyed as a result of having wasted many years on ineffective DMDs. Now I have to wait for the "hope" of remyelination, which we all know is an illusion as it will do nothing in progressive MS with axonal loss. 
  58. There are very few treatments that can help patients with PPMS like HSCT but unless their usage is put into normal practise and easily accessible people are dying while waiting.
  59. My daughter who had MS now for 16 yrs in that time she has been on numerous medicine that has not help at all she steady declining we have made up our minds to go to Russia next year but it be a lot easier for her and us if it was available in Australia and WITHOUT A REFERRAL from a neurologist 
  60. I think the more your quality of life decreases the more willing you are to take the risk.
  61. The chance for some years of a more normal life is worth it!
  62. I had HSCT in Moscow 2013. I was diagnosed PPMS. No enhancing lesions on all MRI tests. Progressing from running half marathons to needing a cane after walking a quarter mile. All within 2 years. Post HSCT I do not need a cane at all and can walk 3 miles with a rest in the middle for about 10 minutes.
  63. Should be offered to people with PPMS as well; there is currently no therapies that are offered for this type of ms. I believe HSCT would stop the progression of PPMS as it does with RRMS. I just wish someone would throw us a bone who are dealing with PPMS.
  64. I have PPMS and had HSCT in Moscow, Russia Aug/Sept 2013. It has stopped my progression and saved my life. You can see my story at I stopped using my trekking pole after 6 months, and can now ride my bike 10 miles, and pushing for longer rides. My gait has improved, as well as toe drop. Before HSCT I could barely walk 1/4 mile, I can now walk 2 miles. So, my quality of life has greatly improved with HSCT. I think it should be an option for anyone with MS, including PPMS/SPMS. I knew the chances of stopping the progression were less for me, than someone with RRMS, but I took the chance. So glad I did!!!
  65. As noted, I'm the spouse, but my husband's neuro has been too conservative all along - "not approved!" "not enough data!" and now my husband is 41 with an EDSS of 7.5-8. Neuro's need to DO MORE and UNDERSTAND that there is a COST to not doing anything or being conservative. 
  66. I'm PPMS,with out hsct there is no other options. Regardless hsct is the only proven treatment to benefit any type of ms,and some forms have great odds. Either way I had no option prior. Now I have hope and a chance. 
  67. Coincidentally, I'm making the choice between HSCT and Lemtrada right now. The more I read about Lemtrada, the more concerned I get about safety and efficacy (particularly among patients who have been on Tysabri before and/or have been diagnosed for 6+ years).
  68. I have CIDP, not MS, but have become friends with many people with MS through various programs. I have seen so many of them deteriorate in the past 5 years. Like me, they started out with canes or walkers when we started the program together 5 years and now most of them are in powerchairs and some are housebound. If they had had a treatment that could stop MS progression they would have some quality of life. 
  69. No drug has yet proven to be as successful as hsct at stopping the progression of ms. I have rrms, i had hsct; the disease was stopped, i got my life back. No chemo = no cure. Hsct is not a new treatment; there is plenty of evidence pointing to its efficacy.
  70. I'm PPMS and have got my name down for Moscow in 2016.
  71. As far as I'm concerned it's a absolute no brainer.
  72. I'm already taking risks by taking Tysabri.
  73. The health departments all around the world should a public (only suffers of diseases that would benifit) referendum and THEN whatever is our wish respect it!!!! WE ARE SUFFERING NOT THEM! I need hsct and I can't afford it. I will be a guinea pig if needed. 
  74. I am RRMS since 4.5 years, 44 years old and mother of 3 girls 5, 9 and 12 years old. I hate taking DMD's because of side effects and hate the uncertainty of thr decease itself and are therefore going to have HSCT to stop this beast. My currently EDSS is 2 and I want to keep it that way or better.
  75. I have been treated 1 year ago I had Fulminant MS which does not respond to any 1st or 2nd line treatments. I did not have time to fail one so I went overseas to be treated. It has been a long road to recovery as I have had to learn to feed myself again, talk again (still a bit slurred) walk again etc etc. I was told I was going palliative weeks before I received treatment. I was having multi-system failure.
  76. No one has actually done a comparison (that I can find) between the deaths occurring for BMT for autoimmune only it seems to be all linked in with the cancer treatments. 
  77. For some reason the use of Tysabri and its links to some 560 deaths from December last year seem to be passed over as a slight inconvenience by most neurologists. 
  78. I met a person about 12 weeks ago that had been on Tysabri for 4 years and other than the initial tests for JCV for which he was positive no further titre level tests had been taken he has since demanded this and has been found to be at a very high level he now has no where to go. 
  79. I know that the trial arm for Dr Burts Satellite trials (Sheffield being one of them in the UK) have a trial arm of Tysabri it is my understanding that if you go JCV+ you are then treated with HSCT. There is no risk of GVHD with Autoglous SCT but a higher risk between myelo and non-myelo the risk is a no brainer when you have aggressive disease.
  80. We should have the evidence for HSCT before it gets offered as a standard of care treatment for MS. 
  81. I chose to have hsct 3 months after diagnosis
  82. I have had HSCT for SPMS & have seen many improvements. Now able to do things that I have not been able to do for many years. If I had done this treatment years ago things would be better, regardless I am glad I did this treatment in Moscow and would have no hesitation recommending this treatment. 
  83. If it can be done daily for other conditions I don't understand why it MS can't be included. Also, I don't unde why all focus is on RRMS & not other types. 
  84. I am not sure about HSCT. I want it to be approved in Australia before i do it.
  85. I'm a 38yr old single father of a 16 yr old daughter when I was first diagnosed I had rapidly progressive MS and I would love too slow some of the progression down or try reverse some of the progression. So I'm willing to try just about anything and I would love to try this not only for myself but my daughter and everybody else that has MS to see if it will help. I'm not sure if you actually reads what is commented on these things but I hope you do. I would love to get a response from you to know that you actually read this. My name is Alan Carpenter my email address is APC 2612 @ 
  86. I was one of the highly active ms'rs in Canada.. I had Malignant MS I was willing to take any risk as I was dying anyway. I am a true believer in HSCT I was a 9.0 at my worst. I now walk, talk, feed myself, type this to you with fingers that once didn't work.
  87. I am a 42 year old woman with SPMS and I had HSCT in Moscow in October 2014. I had no active lesions but was continually declining, I have already had improvement. I was on Tysabri for 2 years prior and declined whilst on the treatment.
  88. I was diagnosed in 1991 and had 19 years of remission. In 2010, I came out of remission, and M.S. Became progressive. Over the next four years I went from teaching couples dance classes with my husband to a cane and a rollator in March 2014. By that time I had learned of HSCT and been accepted by Dr. Fedorenko's in Russia. I was declined by the trials in Seattle and Chicago. I see some minor improvements since returning in August 2014 and am grateful to have been accepted and treated by Dr. F. Even if I don't experience further improvements, I still know I've taken the only treatment that might halt MS.
  89. HSCT beats Lemtrada hands down why not just push HSCT?
  90. Im not sold of the HSCT I am very interested in getting Lemtrada as it is available (almost). I do not want to travel to another country to have HSCT when I can easily get Lemtrada. I put my faith in the neuro to suggest my options, he believes the risks of Lemtrada outweigh the risks of HSCT.
  91. I am RR/SP MS, 2Neurologists are unsure as my relapses and progression are not typical, progression has been relatively fast since diagnosis in 2010. I am on my 3rd DMD and still declining. I want a chance to halt and if I am lucky improve some of my sympoms before I end up in a wheelchair, which I think is likely to happen in the very near future. 
  92. I had hsct and am 100% better than I was.
  93. HSCT should be available in UK, even if MSers have to pay for it. 
  94. 15 Years RRMS, 8 Years SPMS. I'm desperate just to stop the slide!
  95. Ms is a death sentence in itself.
  96. I would without a doubt choose hsct
  97. There are tx in other countries we should be able to evaluate and have access.
  98. It appears thet have success but then we have no real data.
  99. I have MS and if I'm willing to pay for HSCT. I can't understand why I cannot have it in my own country.
CoI: multiple

Labels: , ,